Sunday, November 19, 2017

Kids Helping Kids: 5-year old Singing Sensation Kaitlyn Maher To Perform To Help 5-year old Twins Who Have Lost Their Voices From Dementia

July 30, 2009 by  
Filed under Featured Stories

Five year old singing sensation Kaitlyn Maher, who became the youngest person ever to make it into the Top 10 as a Finalist on NBC’s hit show, America’s Got Talent, will be coming to Reno, Nevada, October 3, 2009 to be the opening act at the Grand Sierra Resort benefiting the The Addi and Cassi Hempel Fund.

Kaitlyn will be performing to help raise money for Addi and Cassi Hempel, 5 year old identical twins, and other children around the world who are afflicted with a rare and fatal childhood dementia called Niemann Pick Type C.  Addi and Cassi and many other children like them can no longer talk or sing their favorite songs due to their deadly illness.

The event called “An Evening of the Stars,” will also include performances by Nevada Entertainer of the Year and master musical impressionist Greg London of the hit show “ICONMAN” and Paul Salos, world famous Frank Sinatra impersonator who like Kaitlyn was also a 2008 finalist on America Got Talent.

Kaitlyn has performed live on the nationally televised 2008 Lighting of the National Christmas Tree ceremony at the White House and at the 2009 National Cherry Blossom Festival chaired by First Lady Michelle Obama. She has also sang the National Anthem and “God Bless America” at the Washington Nationals Major League Baseball game.  In addition to being one of the headlining acts at the McDonald’s Christmas Parade in Orlando, FL, she has appeared on the Today Show, Access Hollywood and was featured in People magazine.

Currently, Kaitlyn is working on a CD for release in the fall of 2009 and recently finished recording her first Disney role in the upcoming movie Santa Buddies scheduled for release during the Christmas holidays later this year.

In 2008, The Addi and Cassi Hempel Fund event drew over 1,000 people and helped raise close to $500,000 dollars for research into “Childhood Alzheimer’s.”

Tickets for “An Evening of Stars” 2009 are on sale now and can be purchased online.

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Nevada Entertainer of The Year and ICONMAN Star Greg London To Perform For Kids Striken By Childhood Alzheimer’s

July 30, 2009 by  
Filed under Featured Stories

 

Greg-London-ICONS-Nevada

Greg London, the master musical impressionist who pays hilarious homage every night to scores of today’s top icons will be bringing his popular and critically acclaimed show "ICONMAN" to the Grand Sierra Resort in Reno, Nevada, on Saturday, October 3, 2009. London will be giving a special performance at "An Evening of Stars," a major fundraising benefit organized by friends and family of 5 year old Reno twins, Addi and Cassi Hempel.

The identical twins are stricken with a rare and fatal cholesterol disease called Niemann Pick Type C, otherwise known as "Childhood Alzheimer’s." Addi and Cassi Hempel’s battle against cholesterol and dementia has been featured on DATELINE, Good Morning America and covered by The Wall Street Journal as discoveries into their cholesterol disease may lead to understanding of illnesses that could impact millions of people.

Nevada Magazine recently named London Nevada Entertainer of the Year 2009 and “ICONMAN” is one of the longest running shows ever at Harrah’s Reno. Harrah’s has graciously allowed London to perform his hit show at the Grand Sierra Resort Ballroom for one night as the venue can accomodate a large audience.

Last year, The Addi and Cassi Hempel Fund’s Moonlight Garden Gala drew over 1,000 people to the Downtown Reno Ballroom including world renown scientists and local dignitaries. Ventriloquist sensation and America’s Got Talent winner Terry Fator and former American Idol singer Melinda Doolittle performed and with the help of the Silver Legacy and scores of local businesses helped raise over $500,000 dollars for research into"Childhood Alzheimer’s."

Purchase tickets for the event online today.

 

 

 

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NIH Announces Therapeutic Trial With Supplement N-Acetyl Cysteine (NAC) To Treat Fatal Children’s Cholesterol Disease

July 13, 2009 by  
Filed under NPC News, Research

NIH-clinical-center

You’ve got to LOVE supplements especially one called N-Acetyl Cysteine (NAC) when it can possibly help treat children afflicted with a rare and fatal cholesterol disease called Niemann Pick Type C (NPC). Considering there has only been only one clinical trial for Niemann Pick Type C ever conducted at the NIH (for a drug called Miglustat/Zavesca that costs about $80,000 per year, per child that is still not approved in the United States) it is quite exciting that we are starting a trial with NAC at a cost of about .20 cents per dose or approximately $350 dollars a year.

NAC is an antioxidant that increases intracellular glutathione. Glutathione issues are a known problem in Alzheimer’s, Parkinson’s and many other neurological conditions. NAC can act as a precursor for glutathione synthesis as well as a stimulator of the cytosolic enzymes involved in glutathione regeneration. What is going on with intracellular glutathione? There must be a common pathway in all of these neurological conditions that is being disrupted (possibly lipids and cholesterol?).

NAC can apparently stop people from pulling their hair out due to a obsessive-compulsive disorder called trichotillomania. Maybe I should try it? I have been pulling my hair out for months over the fact that I know there are more cheap over-the-counter supplements like NAC that could help not only NPC children, but millions of others.

Thanks NIH for helping kids with Niemann Pick Type C!  Our small community can do big things and we’ll make this NAC study a success!

Dear families and supporters of the NPC community,

We would like to inform you of a new therapeutic trial that was approved this week by the NICHD IRB. The name of this study is Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine. This study still has to be reviewed by the FDA, but we are hopeful that we will be able to start enrolling patients in September 2009. Many of you are familiar with the ongoing NPC natural history study at the NIH. As a result of this study, we have identified some promising biomarkers. Some of these biomarkers are related to oxidative stress, which leads to damage in the cells of the body. We would now like to move to the next step and begin to validate the use of these biomarkers. This clinical trial will hopefully lay the groundwork for future therapeutic trials.

This new trial will allow us to both validate these biomarkers and begin to study the ability of N-Acetyl Cysteine (also called NAC) to treat the oxidative stress caused by NPC. NAC is an antioxidant that has been safely used in a variety of medical conditions in both young children and adults. In this study, we will use an effervescent tablet that will be dissolved in water or another clear liquid and taken by mouth three times a day. This will be a blinded, randomized, placebo-controlled cross-over trial. A cross-over trial means that means that each patient will receive NAC for an eight week period and placebo for an eight week period. The placebo does not have any NAC in it, but looks and tastes like the NAC tablet. Blinding means that patients and/or parents will not know the order in which the patient is receiving the NAC or placebo during the study.

We will ask participants to come to the NIH for four outpatient visits over five months. The first admission will be a two day visit and the other three visits will require only one day. Each visit will include a history and physical exam, fasting blood draw, urine collection and a quality of life questionnaire. We will also ask patients to have blood drawn at home and sent to the NIH. Our goal is to have 30 patients complete the trial.

There will also be two “washout” periods of four weeks each, during which neither NAC nor placebo will be given. We will ask patients to discontinue any supplements or medications except for miglustat (Zavesca) or those prescribed by a physician for the treatment of a medical condition other than NPC (such as seizures, ADHD, etc.) for the duration of the study. This is the only way that we can accurately evaluate the efficacy of NAC in NPC. Other supplements such as curcumin, CoQ10 and others could interfere with the study. We just can’t get good data if there are too many uncontrolled variables.

When a new potential treatment becomes available in a rare disease like NPC, it can be very tempting for families to try it on their own rather than in a formal trial, especially one that includes a placebo. One reason for using a cross-over design was to make sure every patient received the drug during the trial. We understand this urge to try these treatments independently. However, if too many parents/patients choose to do this, we will never be able to get good information on drugs that are “off-the-shelf.” This ultimately will make NPC clinical research more difficult and slower.

The NPC Natural History study has been more successful in the past three years than we ever could have imagined. This was only possible with the incredible support from the NPC families that participated and the NPC community as a whole. We will need continued participation to move this effort forward.

Please call or email Nicole Yanjanin at (301) 594-1765 or nyanjanin@mail.nih.gov if you would like more information about the study or if you are interested in participating.

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Wall Street Journal Reports on Cyclodextrin, NPC, and HIV-AIDs

July 4, 2009 by  
Filed under Addi and Cassi News, HIV-Aids

Wall Street Journal Artticle

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